Partners in Progress: Sharing the Vision, Shaping the Future
April 29 – 30, 2015
Sheraton Silver Spring Hotel
8777 Georgia Avenue
Silver Spring, MD 20910
The Patient-Reported Outcome (PRO) Consortium is a public-private partnership established by the Critical Path Institute (C-Path) in cooperation with the U.S. Food and Drug Administration (FDA) and the medical products industry in 2008. The PRO Consortium brings together scientists from C-Path, industry, academia, and regulatory agencies in a pre-competitive environment for the purpose of developing, evaluating, and qualifying PRO instruments for use as primary or secondary endpoint measures in clinical trials designed to evaluate treatment benefit.
On April 29-30, 2015 the SIXTH ANNUAL PATIENT-REPORTED OUTCOME CONSORTIUM WORKSHOP was held in Silver Spring, Maryland. The overall Workshop objectives were to:
- Discuss how all stakeholders can work together to improve patient-focused drug development
- Provide updates on the PRO instrument development and qualification activities within the PRO Consortium’s working groups
- Describe the qualitative and quantitative research that led to development of the Asthma Daily Symptom Diary (ADSD) within the Asthma Working Group
- Discuss the development and implementation of well-defined and reliable clinical outcome assessment (COA) tools for pediatric clinical trials
- Discuss ways to optimize qualitative and quantitative research to more efficiently generate evidence supporting the content validity of new PRO instruments
The following Workshop Agenda provides an overview of the day-and-a-half-long meeting as well as links to the slide sets and posters presented.
Workshop Agenda – Day 1
April 29, 2015
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7:30-8:30 am |
Registration and Continental Breakfast – Cypress BallroomDay 1 Morning
Moderator: Ashley F. Slagle, MS, PhD – Clinical Outcome Assessment (COA) Qualification Scientific Coordinator and Endpoint Reviewer, Study Endpoints and Labeling Development (SEALD), Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA) |
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8:30-8:45 am |
Welcome and Patient-Reported Outcome Consortium Update Stephen Joel Coons, PhD — Executive Director, Patient-Reported Outcome (PRO) Consortium, Critical Path Institute (C-Path) |
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8:45-9:45 am |
Session 1: Stakeholder Collaboration to Improve Patient-Centered Drug Development Moderator: Ashley F. Slagle, MS, PhD – COA Qualification Scientific Coordinator and Endpoint Reviewer, SEALD, OND, CDER, FDA Presenters and Panelists: Janet W. Maynard, MD, MHS – Clinical Team Leader, Division of Pulmonary, Allergy, and Rheumatology Products (DPARP), OND, CDER, FDA Elektra Papadopoulos, MD, MPH – Acting Associate Director, Study Endpoints Team, SEALD, OND, CDER, FDA Katarina Halling, MSc – Patient Reported Outcomes Group Director, AstraZeneca and Industry Co-Director, PRO Consortium |
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9:45-10:10 am |
Session 2: COA Qualification and Study Endpoints Update Presenter: Ashley F. Slagle, MS, PhD – COA Qualification Scientific Coordinator and Endpoint Reviewer, SEALD, OND, CDER, FDA |
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10:10-10:30 am |
Break – 20 min |
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10:30-11:15 am |
Session 3 – The Asthma Working Group: On the Path to Success Moderator: Josephine M. Norquist, MS – Patient-Reported Outcomes Specialist, Merck Sharp & Dohme, Corp Presenters and Panelists: Linda Nelsen, MHS – Director, Patient Reported Outcomes, GlaxoSmithKline Adam Gater, MSc – Director, Endpoint Development and Outcomes Assessment, Adelphi ValuesElektra Papadopoulos, MD, MPH – Acting Associate Director, Study Endpoints Team, SEALD, OND, CDER, FDA |
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11:15-12:15 pm |
Session 4: Patient-Centric Endpoints in Oncology Moderator: Katarina Halling, MSc – Patient Reported Outcomes Group Director, AstraZeneca and Industry Co-Director, PRO Consortium Presenters and Panelists: Cindy Geoghegan – Patient Advocate and Principal, Patient and Partners LLC Paul G. Kluetz, MD – Acting Deputy Director, Office of Hematology and Oncology Products (OHOP), OND, CDER, FDA Ethan Basch, MD, MSc – Director, Cancer Outcomes Research Program, University of North Carolina at Chapel Hill Q & A |
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12:15 – 1:15 pm |
Lunch – Elm I, Elm II and Magnolia Ballroom |
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Day 1 Afternoon Moderator: Katarina Halling, MSc – Patient Reported Outcomes Group Director, AstraZeneca and Industry Co-Director, PRO Consortium |
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1:15 – 2:15 pm |
Session 5: Optimizing Qualitative and Quantitative Research: How to Make the Process More Efficient
Moderator: J. Jason Lundy, PhD – Principal, Outcometrix Presenters and Panelists: J. Jason Lundy, PhD – Principal, Outcometrix Stacie Hudgens, MA (AbD) – Strategic Lead, Quantitative Science, Clinical Outcomes Solutions R.J. Wirth, PhD – Managing Partner, Vector Psychometric Group, LLC Wen-Hung Chen, PhD – Reviewer, Study Endpoints, SEALD, OND, CDER, FDA Q & A |
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2:15 – 3:15 pm |
Session 6: Interpreting Change in Scores on COA Endpoint Measures Moderator: Cheryl D. Coon, PhD – Director, Healthcare Analytics, Adelphi Values Presenters and Panelists: Joseph C. Cappelleri, PhD, MPH, MS – Senior Director of Biostatistics, Pfizer Inc.Cheryl D. Coon, PhD – Director, Healthcare Analytics, Adelphi Values Scott Komo, DrPH – Senior Statistical Reviewer, Office of Biostatistics, CDER, FDA Laura Lee Johnson, PhD – Associate Director, Division of Biometrics III, Office of Biostatistics, Office of Translational Sciences, CDER, FDA Q & A |
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3:15 – 3:45 pm |
Break – 30 min |
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3:45 – 4:45 pm |
Session 7: Thinking with the End in Mind: From COA Instrument to Endpoint Moderator: Jean Paty, PhD – Principal Advisory Services, Quintiles Presenters and Panelists: David S. Reasner, PhD – Vice President, Data Science and Head, Study Endpoints, Ironwood Pharmaceuticals Laura Lee Johnson, PhD – Associate Director, Division of Biometrics III, Office of Biostatistics, Office of Translational Sciences, CDER, FDA Elisabeth (Liz) Piault-Louis, PharmD, MA – Principal Outcomes Research Scientist, Oncology Genentech, a member of the Roche Group Q & A |
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4:45 – 5:15 pm |
Day 1 Closing Remarks, Day 2 Preview Adjourn |
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5:30 – 7:00 pm |
Reception and Poster Session – Magnolia Ballroom Asthma Cognition Depression Functional Dyspepsia Irritable Bowel Syndrome (IBS) Non-Small Cell Lung Cancer (NSCLC) |
Workshop Agenda – Day 2
April 30, 2015
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7:30-8:30 am |
Registration and Continental Breakfast – Cypress Ballroom |
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8:30 – 9:00 am |
Session 8: Results from “Literature Review to Determine Empirical Basis for Response Scale Selection in Patient-Reported Outcome Instrument Development” Presenter: Katharine S. Gries, PharmD, PhD – Senior Research Associate, Evidera Q & A |
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9:00 – 10:00 am |
Session 9: Practical Considerations in Implementing a Pediatric COA Measurement Strategy: A Case Study in Functional Constipation
Moderator: Sarrit Kovacs, PhD – Study Endpoints Reviewer, SEALD, OND, CDER, FDA Presenters and Panelists: Andrew E. Mulberg, MD, FAAP, CPI – Deputy Director, Division Gastroenterology and Inborn Errors Products (DGIEP), OND, CDER, FDA Diane Turner-Bowker, PhD – Engagement Leader I, Quintiles Gina Calarco (Smith), MPH, RN, CCRC – Associate Director and Deputy Head of the Pediatric Center of Excellence, Quintiles Jean Paty, PhD – Principal Advisory Services, Quintiles Q & A |
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10:00 – 10:30am |
Break – 30 min |
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10:30 – 11:30 am |
Session 10: ePRO Science and Innovation: BYOD Approaches and Equivalence Across Administration Modalities Moderator: Susan Vallow, RPh, MBA, MA – Senior Director, Patient Focused Outcomes, GlaxoSmithKline Presenters and Panelists: Willie Muehlhausen, DVM – Vice President, eCOA and Innovation, ICON plc Chad Gwaltney, PhD – Chief Scientist and Regulatory Advisor, Endpoints, ERT Virginia (Gini) Kwitkowski, MS, RN, ACNP-BC – Clinical Team Leader and Associate Director for Labeling, Division of Hematology Products, OHOP, OND, CDER, FDA Cindy Howry, MS – Vice President, Product Strategy and Innovation, YPrime and Vice Director, ePRO Consortium Sheila Rocchio, MBA – Vice President, Marketing and Strategy, PHT Q & A |
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11:30 – 12:15 pm |
Session 11: The Relevance of Patient-Reported Endpoints to Payers and Regulators: Is there Common Ground? Moderator: Peter C. Trask, PhD, MPH – Principal Scientist, Patient Centered Outcomes Research, Genentech Presenters and Panelists: David S. Reasner, PhD – Vice President, Data Science and Head, Study Endpoints, Ironwood Pharmaceuticals Vasudha Bal, MSc, MBA – Director, Patient Reported Outcomes, Novartis Pharmaceuticals Corporation Katarina Halling, MSc – Patient Reported Outcomes Group Director, AstraZeneca and Industry Co-Director, PRO Consortium Selena R. Daniels, PharmD, MS – Study Endpoints Reviewer, SEALD, OND, CDER, FDA Alan L. Shields, PhD – Vice President, Endpoint Development and Outcomes Assessment, Adelphi Values Robin S. Turpin, PhD – Director and Head, HEOR, U.S. Medical and Scientific Affairs, Takeda Pharmaceuticals, USA Q & A |
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12:15-12:30 pm |
Closing Remarks |
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12:30 pm |
Adjourn |
